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Neurogene Receives FDA Approval for Gene Replacement Clinical Trial for Rett Syndrome

January 23, 2023

When we make announcements at RSRT about progress I’m careful to match my tone to the significance of the news. Many advancements, while certainly important, are early steps on the road to potential treatments or cures, and my words reflect that. But there are rare times when I have reason not to hold back on my enthusiasm. Today is one of those times.

Neurogene has announced that its Investigational New Drug (IND) application for a gene replacement clinical trial for Rett syndrome has been accepted by the FDA. This is a moment my colleagues and I have been working towards and looking forward to for a long time. It’s a moment that our supporters and the families that fundraise for us should look to as proof positive that their generosity and efforts truly make a difference. It’s a moment that I hope all Rett families will take stock in as confirmation that the research really is progressing, and that your involvement with us at RSRT matters.

The Neurogene program is close to our hearts at RSRT. In fact, it’s unlikely that this clinical trial would be happening were it not for RSRT. This trial is the result of many years of research by Stuart Cobb, PhD. He was a member of our Gene Therapy Consortium and the Cobb lab at the University of Edinburgh received $3 million of funding from RSRT during the last decade. In 2018, RSRT’s chief medical officer, Randy Carpenter, MD, and I introduced him to Neurogene’s founder and CEO, Rachel McMinn, PhD. The rest, as they say, is history. Dr. Cobb became chief scientific officer of Neurogene and was able to focus the full resources of the company on accelerating a gene replacement for Rett.

This is a big iron in the fire in the fight against Rett syndrome, and I am excited and optimistic. But our work is far from done. We must continue advancing all of our genetic-based strategies while simultaneously facilitating the involvement of additional biopharmaceutical companies. We won’t rest at RSRT until Rett is eradicated.

Congratulations to everyone at Neurogene on this milestone! On behalf of Rett families around the world, we thank you for taking on Rett syndrome!

I encourage you to read Neurogene's press release and letter to the Rett syndrome community. See links below.

Monica Coenraads